Towards gene therapy for Angelman Syndrome using artificial transcription factors
Dr. Segal has significant experience in synthesizing molecules that bind to DNA and are able to regulate gene activity. ASF has provided funds to this investigator to pursue the possibilities that such agents, called artificial transcription factors, could have an effect of increasing the expression of paternal UBE3A. These transcription factors could operate either very close to UBE3A or in regions some distance away, for example where the imprinting control center is located. To date, although preliminary work was encouraging, no viable transcription factor has been identified that brings about significant activation of the otherwise silenced UBE3A.